Prevail Therapeutics Receives European Commission Orphan Designation for PR006 for the Treatment of Frontotemporal Dementia

Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the European Commission has granted orphan designation for PR006 for the treatment of frontotemporal dementia (FTD). PR006 is an investigational AAV9 gene therapy delivering the GRN gene and is being developed as a potential therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

“The European Commission’s decision to grant orphan designation for PR006 is an important step in helping to advance this potential therapeutic option for patients with frontotemporal dementia with GRN mutations,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “We are excited to progress clinical development of PR006 as part of our mission to deliver a potentially disease-modifying gene therapy to these patients as quickly as possible.”

The Company expects to initiate enrollment in the Phase 1/2 PROCLAIM trial of PR006 in the fourth quarter of 2020, and it currently anticipates it will provide a biomarker and safety analysis on a subset of patients in the PROCLAIM trial in 2021.

The U.S. Food and Drug Administration has also granted Orphan Drug designation for PR006 for the treatment of FTD and Fast Track designation for FTD-GRN.

About European Commission Orphan Designation
Orphan designation is granted by the European Commission to encourage development of medicines intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the European Union. Orphan designation by the European Commission provides companies with certain benefits and incentives, including protocol assistance, reduced regulatory fees and 10 years of market exclusivity following regulatory approval.

About Prevail Therapeutics
Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

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